It’s time to move on.

I’ve grown old in this MPN world.

In the past 20 years, on my way to myelofibrosis, I’ve lost good friends to this disease, people I loved, men and women I miss. And I suffered the uncertainties and pain of many others. On behalf of patients, I fought several ferocious battles with self-styled experts, bureaucrats, drug makers and hematologists.

I need to stop losing friends and fighting losing battles.

MPNforum – volunteers, writers, physicians — helped bring us together, let us see one another’s faces and hear our stories, helped present the unfolding science. We reported the good and bad of the Jakafi story and drug pricing, started the Fatigue Project dialog, created the Zebra Coalition for patient advocacy, uncovered the hidden Sanofi story, created the MPN Quarterly Journal, published the Interferon Papers and four dozen monthly issues in the past four years plus special reports.

If that all reads like an obit, perhaps it should. It’s time to move on. [Read more.]

The MPN patient’s dilemma

The attempt by a group of investors to gain rights to Sanofi’s fedratinib brings the MPN patient’s dilemma into sharp focus. That report surfaced after Zhaohui Ye at Johns Hopkins demonstrated the ability of gene editing technology to clip a JAK2 mutation out of a human stem cell.

These two events – bringing back Sanofi’s failed JAK2 inhibitor and opening a genetic path to an MPN cure — are the prism through which we can clearly see the clash of the past and future. And the root of our central dilemma as MPN patients.

Today, a decade after the JAK2 discoveries, the landscape of MPN therapy has expanded but remains fundamentally unchanged. All around us we see both the benefits and limitations of treatment aimed at relieving the effects of our MPNs while leaving the underlying disease unchecked. Belief in a Magic Pill has proven to be a deadly delusion. [Read More]

The season of big, significant genome editing conferences rolls on this month with the IBC Genome Editing Applications meeting in Boston’s Back Bay. This one, the second big meeting to be held in Boston this storm-tossed season, is aimed squarely at therapeutic and biomedical applications of gene editing. — including CRISPRs. The agenda directly addresses the concerns of the MPN blood cancer community. The conference takes place March 18 and 19.

Presenters and panels include genomic top guns running academic and commercial research labs, bench scientists and some of the founders of CRISPR technology, including Feng Zhang, PhD, the award-winning young scientist from the Broad Institute of MIT and Harvard. The meeting is another vital overview from the frontlines of the genomic revolution moving much faster than anyone anticipated.

How fast? Here’s the abstract for keynote speaker, Harvard’s George Church’s presentation on Radical Genome Editing:

“Our ability to view and alter biology progresses exponentially, outpacing electronics — synthesizing billions of base pairs of DNA on chips and genome editing millions of new organisms per day — moving from correlational to causality studies…”

Remember that big survey the folks at the Mayo Clinic Survey Research Center released last year via on-line MPN media?

The results are now headed to the coming European Hematology Association meeting for poster representation by the Mayo Clinic’s Dr. Robyn Scherber and Dr. Ruben Mesa.

Dr. Robyn Scherber

Robyn and her crew reviewed analyses of the results of this 70 item study and the nearly 1800 responses in multiple areas – primarily efficacy of interventions to alleviate fatigue and closer scrutiny of the nature of MRF (myeloproliferative related fatigue).

Their conclusions are leading to that promised third phase interventional study where specific agents and techniques to relieve MRF will be explored. Stay tuned.

“I am a 5-year bone marrow transplant survivor, having been actively treated for myelofibrosis, one of the MPNs, during the four years prior to transplant.

In late 2013 I was nominated to join a peer review panel of scientists evaluating grant applications for cutting edge, “outside of the box” medical research into bone marrow failure diseases similar to our MPNs. While I don’t have any formal education in medicine, I quickly found out that my past experience as a patient with myelofibrosis and my experience in the MPN community..[More] – by Marty Prager

.Genetic engineers, researchers, biochemists

$200,000 Research Grants
available from the MPN Research Foundation for
research into gene editing applied to MPNs.
Information here.
Application here:
Deadline to apply: April 1

The Spring MPN Quarterly Journal will focus on CRISPR Cas9 and gene editing.